Time in a bottle

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Expensive new cancer drugs are extending
lives by as much as three years. But what
price tag should we place on prolonged
life? Lisa Mitchell reports.

Graham Cook has more serious
issues to nurse than pity, but as
we sit on a park bench on a
glorious afternoon, he covers
his clear blue eyes with his
hands. Emotion takes over as he
speaks of the oncologist who
has, so far, managed to keep him alive. "He is
a miracle man."

Cook has an extremely rare disease called
Hypereosinophilic Syndrome (HES). His
treatment, Glivec, is a new wonder drug or
"targeted therapy" that is the latest addition
to the arsenal for cancer treatment. Targeted
therapies offer prolonged quality of life,
rather than a cure, but there are two major
obstacles — access and affordability — with
treatments costing as much as $50,000 a
year. For Cook, and others like him, the
question is: How much am I willing to pay
for borrowed time? The answer for Cook is
$12,000 a year.

Cook, 57, is a former self-employed
accountant who now works two days a week
for a housing company. It's all he can
manage, he says, as he pulls a piece of paper
from his pocket. It is a list of 16 drugs that he
swallows daily. "I can't do complicated jobs
any more. I find it very hard to think
straight."

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At the time of his diagnosis in 1995, his
doctors knew of no effective treatment for
HES. Cook spent four-and-a-half years on
chemotherapy until it began to kill off the
nerve endings in his feet. Unable to walk
properly, he suffered a series of falls and
fractured his back, broke a hip and ripped
the muscles from his kneecap. His bones
had become brittle because of intravenous
steroid treatment that also caused diabetes
and blood clotting in his heart. At that point,
he expected to die.

Then his doctor, Associate Professor Jeff
Szer, a hematologist at the Royal Melbourne
Hospital, came across Glivec, a targeted
therapy that had produced astounding
results in US clinical trials. Within a week of
starting treatment, the telltale signs of Cook's
HES — excessive eosinophil cells in the
blood — had decreased dramatically. He no
longer needs chemotherapy and the nerves
in his feet have almost recovered.

Ethically, how does an oncologist deal with a patient whose potential lifespan may be beyond their financial means?

Cook's private health insurer, Australian
Unity, agreed to pay for eight months of
treatment but has declined his request to
fund it further. He is on his own.

"There's no point in being pessimistic,
only positive," he says. "I can't do anything
like I used to, but I'm working, aren't I?" He
says he will manage to pay the $12,000
because he has no choice. He has already
sold his home and moved to a smaller one.

"My wife and I regard it as the top priority.
It's the way I can live . . . I can't go back to
where I was."

Carole Sowerby's $45,000 treatment is free while she remains on a clinical trial.Picture:Simon Schluter

Targeted therapies offer a vastly
different approach to cancer
treatment, says Dr Grant McArthur,
an oncologist at the Peter MacCallum
Cancer Centre. These precision drugs
target the specific proteins in cells that cause
normal cells to become cancerous. The
results can be startling and with far fewer
side effects than chemotherapy or
radiotherapy. It can mean a pill a day instead
of invasive surgery.

Unlike standard treatments, targeted
therapies seem to suppress or "hold" a
tumour, or produce a slow shrinkage. Some
return severely affected blood counts to
near-normal, but what works for one person
may not work for another and the treatment
may work for a period of time and then
cease to be effective. In spite of the
technological advance, targeted therapies
certainly don't hold all the answers.

Carole Sowerby was diagnosed with the
rare CMML (chronic myelomonocytic
leukaemia) last Easter. Her spleen was
enlarged so that she looked pregnant and
her weight dropped from 48 kilograms to 39
kilograms. Sowerby did not expect to be
around to celebrate last Christmas. But after
just one week on Glivec, she had improved
markedly.

"My white cells were supposed to be
between four and 10. Mine were up to 101,
but as soon as I started Glivec, it just went
down, until three months later there were
hardly any leukaemia cells at all. My body
went back to normal, my spleen went down,
it was like I never had it."

Sowerby is one of the lucky ones. Her
Glivec treatment, which would normally cost
$45,000 a year, is free while she remains on a
clinical trial. After that, she does not know
how she will pay for it. "I don't worry about
the future, I think the future has a way of
looking after itself. I just get on with things."

Glivec is the most significant targeted
therapy success story in Australia. It has
extended the life of some patients by as
much as three years on the expected
prognosis, but there is not yet enough data
to know whether Glivec's effect will be
lasting.

Another winner seems to be Herceptin,
which works on 10 to 15 per cent of women
with breast cancer. About 65 per cent of non-Hodgkins Lymphoma sufferers can now
turn to Rituximab for hope and oncologists
are eagerly awaiting the results of a local
clinical trial testing Avastin on bowel cancer.

Avastin is known to extend median survival
by five months and with none of the side
effects of chemotherapy, such as hair loss
and vomiting.

"The important thing is that this is just
the beginning," says McArthur. "We've got so
many of these types of treatments coming
forward, we've really got to get these issues
out for public discussion so we can come up
with a process that allows patients to access
them."

If targeted therapies offer time in a bottle,
what price tag should we place on
prolonged life? Ethically, how does an
oncologist deal with a patient whose
potential lifespan may be beyond their
financial means? These are the questions
that trouble oncologists as they confront
ever-hopeful patients.

Herceptin can prolong survival in some
patients by five months, says Dr Richard
Bell, a senior clinical adviser for the Cancer
Council of Victoria and director of cancer
services at Geelong Hospital.

"I put it to you
that for the woman concerned, that might
be a child graduating from high school or
university, it might be another wedding
anniversary, it might be a grandchild born.
There could be a lot of significant
milestones."

Bell would like to get his hands on
Velcade, which is available in the United
States for the treatment of multiple
myeloma, a cancer of the blood. In Australia,
Velcade and other unapproved targeted
therapies can be imported under special
conditions set by the Therapeutic Goods
Administration (TGA) for individual
patients.

"But the cost is £3220 ($A7790),
which is sufficient for three weeks'
treatment, which you would do up to eight
times," he says.

Glivec can cost up to $45,000 a year,
depending on the type of cancer it treats,
while Iressa, the wonder drug for lung
cancer, can cost as much as $50,000 a year.

Oncologists are bound in an ethical
straightjacket by these issues. Says Dr Peter
Gibbs, an oncologist at the Royal Melbourne
Hospital who is involved with the Avastin
trials: "How can you judge one patient being
more worthy than another? Should you offer
an expensive treatment to a patient, not
knowing their financial status, and risk the
distress it may cause if they can't afford it?
Some people would pay $40,000 for an extra
three months of life. Others don't want to
take that money away from their wife and
kids . . . It's an incredibly complex situation."

But targeted therapies can also cost as
little as $24 a month when they are approved
for the Pharmaceutical Benefits Scheme
(PBS), which brings us to the other big issue.
Bell likes to call it "the enormous inequity of
access".

Hog-tied by red tape, some oncologists
are resorting to "creative measures" to care
for their patients. Their frustration has to do
with Australia's highly selective and lengthy
approval process for drugs, which must be
registered with the TGA and then apply for
Government subsidy on the PBS.

Glivec, for example, was registered by the
TGA but took 12 months to gain approval for
a PBS listing. But it is only registered with
the TGA for the treatment of two relatively
uncommon cancers — gastrointestinal
stromal tumour (GIST) and chronic
myelogenous leukaemia — because drug
companies must provide costly data to prove
Glivec's effectiveness for each new cancer
treatment.

Even now, not all GIST patients
may access it, explains Gibbs. According to
the TGA guidelines for GIST treatment,
tumours must reduce by 50 per cent to be
eligible for further funded treatment.
Patients whose tumours shrink slightly or
remain stable are not eligible.

"It's a very difficult position for
oncologists, to give patients a successful
treatment and then have to take it away,"
says Gibbs.

Several oncologists, who asked not to be
named, feel compelled to work around the
system. They might massage paperwork to
ensure the ongoing treatment for patients,
or use the remaining drug supply of a
patient who has died to treat others. Some
use drugs that are nearing their expiry date.

Once targeted therapies are TGA-approved,
the drug companies often provide
free samples to patients as they seek a PBS
listing, but their compassion usually cannot
match the length of the approval process.

The US drug market is their best hope of
recouping research and development costs,
being 50 per cent of the global
pharmaceutical market, while Australia
captures only 1 per cent of the market.

Competition to list on Australia's PBS is
tough, especially for costly targeted
therapies. The scheme is already under fire
for its soaring $5 billion operating cost.

Oncologists argue that targeted therapies
could be accommodated on the PBS if the
Government could rein in the listings and
the over-prescribing of multiple drugs for
common conditions such as cholesterol,
blood pressure, ulcers and arthritis.

A
Government spokesperson for the
Pharmaceutical Benefits Advisory
Committee (PBAC) refutes this, saying the
committee has the appropriate procedures
and checks in place to ensure this does not
happen and that PBS funds are used costeffectively.

PBAC has also reduced the
average time between drugs being
recommended and listed from 9.5 months in
1995 to five months in 2003.

Surely the cost of maintaining cancer
patients in hospital outweighs the cost of
keeping them healthy and at home on
targeted therapy, says Bell, but no one has
the definitive cost analysis data that might
sway PBAC to favour more of these chronic
conditions.

It would be relatively cheap for them to
list rare conditions such as HES and CMML,
argues McArthur. It would probably cost
between $2 million and $3 million per year,
compared to the $50 million per annum it
might cost to treat 3000 new bowel cancer
patients with Avastin.

"But these are new
therapies and there's a learning curve for
everyone involved in making these
decisions. We're hoping, with time, the way
they deal with these drugs will improve," he
says.

As the impassioned debate continues,
even the finger pointers
acknowledge the system depends on
too many parties for one correction
to produce an A-grade result.

The TGA and PBS say they are doing
their best to provide equitable access and
fast turnaround on the applications they
receive. Some hospitals try to fund patients
who are unable to afford treatment.

Drug
companies such as Novartis, which makes
Glivec, will sometimes provide drugs on a
compassionate basis or expand clinical
trials to squeeze in those who are
floundering, but they cannot continue ad
infinitum.

Private health insurers are under
no legal obligation to fund exceptional
medicines, but may opt to do so for certain
cases.

Australian Unity funded eight
months' supply for Graham Cook before it
declined further funding.

"Over a week, we receive on average 10
requests for drugs that may cost from $3500
to $15,000 for one dose, and multiple doses
may be required," says Ann McNair, the
health services manager for Australian Unity
health insurance.

"The ex-gratia payment
bucket that we have for these things is very
limited and we try to give as many members
as possible some money from that bucket . . .
As a society, our Government is in a very
difficult situation to work out where our
health dollar should be effectively spent. It's
human nature to say, I want this money to
be spent on me . . . Then there are those on
public healthcare who don't have any access
to these drugs . . . I really don't know what the
solution is."

Lobbying proved successful for the breast
cancer drug Herceptin, after it was turned
down by PBAC. The breast cancer network
and former health minister Michael
Wooldridge managed to set up alternative
funding to subsidise treatment at $25 a
month, instead of $1000.

For Cook, the issue is double-barrelled.
Glivec is not registered with the TGA to treat
his rare form of cancer. Even if it was, who
would agitate for its PBS approval, apart
from Cook and his doctor?

"We are faced with this in many areas of
medicines where we have a known effective
treatment for a small group of parties with a
rare disorder, but it's almost impossible to
collect the sort of rigorous scientific data
that would be required to satisfy (TGA)
registration and (PBS) reimbursement
criteria," says Szer.

In the meantime, patients like Cook are
left to judge the worth of their own lives. "I
thought, surely, if I write to the minister and
deal with the insurance company, and Dr
Szer deals with the drug company, we'll keep
hitting them on so many fronts that,
eventually, someone will fold. But boy, it's
hard. They just don't . . . Logic tells me that
people can't be that callous, they really can't
be."